Adzynma
rADAMTS13 · Takeda Manufacturing Austria AG
ORPHAN EXCEPTIONAL CIRCUMSTANCES AUTHORISED IN THE EUWhat Adzynma is used for
Treatment of congenital thrombotic thrombocytopenic purpura (cTTP) due to ADAMTS13 deficiency
Approved use, as published by the European Medicines Agency.
What the labels mean
- Orphan medicine: for a rare disease (affecting no more than 5 in 10,000 people in the EU).
- Approved under exceptional circumstances: full data cannot be collected, usually because the disease is very rare.
Key facts
- Brand
- Adzynma
- Active substance
- rADAMTS13
- Disease area
- Purpura, Thrombotic Thrombocytopenic
- Medicine group
- Antithrombotic agents;Enzymes
- ATC code
- B01AD13
- Marketing authorisation holder
- Takeda Manufacturing Austria AG
- Authorised in the EU since
- 2024
Official information
Specialist medicine: started and supervised by a specialist doctor or hospital. This page is a plain-language summary of public official information, not medical advice; follow your doctor and the official product information.
Common questions
What is Adzynma used for?
Treatment of congenital thrombotic thrombocytopenic purpura (cTTP) due to ADAMTS13 deficiency
What is the active substance in Adzynma?
Adzynma contains rADAMTS13.
Who makes Adzynma?
The EU marketing authorisation holder is Takeda Manufacturing Austria AG.
Is Adzynma an orphan medicine?
Yes. Adzynma is designated an orphan medicine in the EU, for a rare disease (Purpura, Thrombotic Thrombocytopenic).
Source: European Medicines Agency medicines data (ema.europa.eu), reproduced with acknowledgement of the source. Page made 5 November 2026 by Satya Babu.
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